AAV vectors show great promise as vectors for gene therapy. We are developing vectors which will be useful to treat rhesus monkeys with globoid cell leukodystrophy. We inoculated 2 infant rhesus monkeys with an AAV vector containing the green fluorescent protein reporter gene intramuscularly and intracerebrally. Biopsies from both animals showed a small amount of fluorescence in muscle. One monkey was euthanatized for tissue collection. A small amount of fluorescence was observed adjacent to the needle tract in the brain. To date, this pilot study has demonstrated the feasability of using an AAV vector in these monkeys. We plan to reinoculate the remaining monkey to evaluate persistence at the original inoculation sites and the effect of a second administration of vector. FUNDING Base Grant, Venture Research PUBLICATIONS None